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CRISPR-mediated generation and characterization of a Gaa homozygous c.1935C>A (p.D645E) Pompe disease knock-in mouse model recapitulating human infantile onset-Pompe disease

Scientific reports, 2022-12, Vol.12 (1), p.21576-13, Article 21576

2022

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Hepatitis C virus has a genetically determined lymphotropism through co-receptor B7.2

Nature communications, 2017-01, Vol.8 (1), p.13882-13882, Article 13882

2017

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Base editing corrects the common Salla disease SLC17A5 c.115C>T variant

Molecular therapy. Nucleic acids, 2023-12, Vol.34, p.102022, Article 102022

2023

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Developments in continuous therapy and maintenance treatment approaches for patients with newly diagnosed multiple myeloma

Blood cancer journal (New York), 2020-02, Vol.10 (2), p.17-17, Article 17

2020

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Characterization of PARP6 Function in Knockout Mice and Patients with Developmental Delay

Cells (Basel, Switzerland), 2021-05, Vol.10 (6), p.1289

2021

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Altered NEP2 expression and activity in mild cognitive impairment and Alzheimer's disease

Journal of Alzheimer's disease, 2012-01, Vol.28 (2), p.433-441

2012

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Hepatitis C Virus (HCV)-Induced Immunoglobulin Hypermutation Reduces the Affinity and Neutralizing Activities of Antibodies against HCV Envelope Protein

Journal of Virology, 2008-07, Vol.82 (13), p.6711-6720

2008

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CRISPR-Cas9 generated Pompe knock-in murine model exhibits early-onset hypertrophic cardiomyopathy and skeletal muscle weakness

Scientific reports, 2020-06, Vol.10 (1), p.10321-10321, Article 10321

2020

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PARP6 is a Regulator of Hippocampal Dendritic Morphogenesis

Scientific reports, 2016-01, Vol.6 (1), p.18512-18512, Article 18512

2016

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Neprilysin-2 Is an Important β-Amyloid Degrading Enzyme

The American journal of pathology, 2011, Vol.178 (1), p.306-312

2011

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Prime editing corrects the c.1826dupA mutation in infantile-onset Pompe disease

Molecular genetics and metabolism, 2022-02, Vol.135 (2), p.S104-S105

2022

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Generation of infantile- and late-onset Pompe disease models using CRISPR-Cas9 genome editing

Molecular genetics and metabolism, 2017-01, Vol.120 (1-2), p.S67-S67

2017

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P4-271: Relative contribution of ER calcium channels to synaptic activity and plasticity in mutant PS1 Alzheimer's disease mouse models

Alzheimer's & dementia, 2008-07, Vol.4 (4), p.T750-T751

2008

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Optimization of CRISPR mediated genome correction of Pompe disease-specific GAA mutations in C2C12 mouse myoblasts

Molecular genetics and metabolism, 2018-02, Vol.123 (2), p.S56-S56

2018

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Genomic correction of Pompe disease knock-in mouse myoblasts via CRISPR-Cas9 homology-directed repair

Molecular genetics and metabolism, 2020-02, Vol.129 (2), p.S143-S143

2020

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CRISPR-mediated generation and characterization of the homozygous c.1935c > a (p.d645e) Pompe disease knock-in mouse model

Molecular genetics and metabolism, 2022-02, Vol.135 (2), p.S54

2022

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CRISPR-mediated generation and characterization of the GAA homozygous c.1935c > a (p.d645e) Pompe disease knock-in mouse model

Molecular genetics and metabolism, 2022-02, Vol.135 (2), p.S54-S54

2022

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CRISPR-Cas9 generated Pompe knock-in murine model exhibits early-onset cardiac hypertrophy and motor impairment

Molecular genetics and metabolism, 2019-02, Vol.126 (2), p.S76-S76

2019

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Characterization of CRISPR-Cas9 generated Pompe disease models

Molecular genetics and metabolism, 2018-02, Vol.123 (2), p.S66-S67

2018

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Longitudinal assessment and immune response to recombinant GAA in CRISPR-Cas9 generated Pompe disease knock-in mice

Molecular genetics and metabolism, 2020-02, Vol.129 (2), p.S75-S76

2020

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F-spondin gene transfer improves memory performance and reduces amyloid-β levels in mice

Neuroscience, 2012-10, Vol.223, p.465-472

2012

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Isatuximab Plus Pomalidomide/Low-Dose Dexamethasone Versus Pomalidomide/Low-Dose Dexamethasone in Patients with Relapsed/Refractory Multiple Myeloma (ICARIA-MM): Characterization of Subsequent Antimyeloma Therapies

Blood, 2022-11, Vol.140 (Supplement 1), p.608-610

2022

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Updates from ICARIA-MM, a phase 3 study of isatuximab (Isa) plus pomalidomide and low-dose dexamethasone (Pd) versus Pd in relapsed and refractory multiple myeloma (RRMM)

Journal of clinical oncology, 2021-05, Vol.39 (15_suppl), p.8017-8017

2021

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A phase III randomized, open label, multicenter study comparing isatuximab, pomalidomide, and low-dose dexamethasone versus pomalidomide and low-dose dexamethasone in patients with relapsed/refractory multiple myeloma (RRMM)

Journal of clinical oncology, 2019-05, Vol.37 (15_suppl), p.8004-8004

2019

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CRISPR-mediated generation and characterization of a Gaa homozygous c.1935C>A (p.D645E) Pompe disease knock-in mouse model recapitulating human infantile onset-Pompe disease

Hepatitis C virus has a genetically determined lymphotropism through co-receptor B7.2

Base editing corrects the common Salla disease SLC17A5 c.115C>T variant

Developments in continuous therapy and maintenance treatment approaches for patients with newly diagnosed multiple myeloma

Characterization of PARP6 Function in Knockout Mice and Patients with Developmental Delay

Altered NEP2 expression and activity in mild cognitive impairment and Alzheimer's disease

Hepatitis C Virus (HCV)-Induced Immunoglobulin Hypermutation Reduces the Affinity and Neutralizing Activities of Antibodies against HCV Envelope Protein

CRISPR-Cas9 generated Pompe knock-in murine model exhibits early-onset hypertrophic cardiomyopathy and skeletal muscle weakness

PARP6 is a Regulator of Hippocampal Dendritic Morphogenesis

Neprilysin-2 Is an Important β-Amyloid Degrading Enzyme

Prime editing corrects the c.1826dupA mutation in infantile-onset Pompe disease

Generation of infantile- and late-onset Pompe disease models using CRISPR-Cas9 genome editing

P4-271: Relative contribution of ER calcium channels to synaptic activity and plasticity in mutant PS1 Alzheimer's disease mouse models

Optimization of CRISPR mediated genome correction of Pompe disease-specific GAA mutations in C2C12 mouse myoblasts

Genomic correction of Pompe disease knock-in mouse myoblasts via CRISPR-Cas9 homology-directed repair

CRISPR-mediated generation and characterization of the homozygous c.1935c > a (p.d645e) Pompe disease knock-in mouse model

CRISPR-mediated generation and characterization of the GAA homozygous c.1935c > a (p.d645e) Pompe disease knock-in mouse model

CRISPR-Cas9 generated Pompe knock-in murine model exhibits early-onset cardiac hypertrophy and motor impairment

Characterization of CRISPR-Cas9 generated Pompe disease models

Longitudinal assessment and immune response to recombinant GAA in CRISPR-Cas9 generated Pompe disease knock-in mice

F-spondin gene transfer improves memory performance and reduces amyloid-β levels in mice

Isatuximab Plus Pomalidomide/Low-Dose Dexamethasone Versus Pomalidomide/Low-Dose Dexamethasone in Patients with Relapsed/Refractory Multiple Myeloma (ICARIA-MM): Characterization of Subsequent Antimyeloma Therapies

Updates from ICARIA-MM, a phase 3 study of isatuximab (Isa) plus pomalidomide and low-dose dexamethasone (Pd) versus Pd in relapsed and refractory multiple myeloma (RRMM)

Carfilzomib Thalidomide and Dexamethasone Is Safe and Effective in the Treatment of Relapsed/Refractory Multiple Myeloma: An Open Label Phase II Australasian Leukaemia and Lymphoma Group (ALLG) MM 018/ Asian Myeloma Network (AMN) 002 Study

A phase III randomized, open label, multicenter study comparing isatuximab, pomalidomide, and low-dose dexamethasone versus pomalidomide and low-dose dexamethasone in patients with relapsed/refractory multiple myeloma (RRMM)

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